P716: OUTCOMES AND PATTERNS OF TREATMENT IN CHRONIC MYELOID LEUKEMIA, A GLOBAL PERSPECTIVE BASED ON A REAL-WORLD DATA GLOBAL NETWORK
Authors: A. Sanz, R. Ayala, G. Hernández, N. López, D. Gil, R. Gil, R. Colmenares, G. Carreño-Tarragona, J. M. Sánchez Pina, R. A. Alonso, N. García Barrio, D. Pérez-Rey, L. Meloni, M. Calbacho, M. Pedrera-Jiménez, P. Serrano-Balazote, J. de la Cruz, J. Martínez-López
Journal: HemaSphere (2022)

Background: The natural history of chronic myeloid leukemia (CML) experienced a major change in the early 2000s with the development of the first BCR-ABL1 specific tyrosine kinase inhibitors (TKIs); however, there is limited supporting evidence from electronic health records (i.e., real-world data [RWD]). TriNetx is a global health research platform that provides researchers access to electronic medical records from healthcare organizations (HCOs) for conducting research studies. It allowed us to analyze trends in survival in CML patients throughout the years from a global perspective, as well as examine relevant comorbidities and long-term toxicities. Aims: The objective of this study is to better define the current survival landscape of CML and the long-term toxicities, so that we can have a better and updated understanding of the disease and the actual clinical practice in local, regional and global settings. Methods: We analyzed data from more than 7500 patients diagnosed with CML from four research networks (Hospital 12 de Octubre (H12O), the EMEA network, the US network, the Global Network) covering more than 16 million subjects. Propensity score matching for age and gender was applied, comparing samples equal in size (CML and controls). This study was conducted with anonymized data accessed via the TriNetX platform, which provided diagnoses, procedures, medications, laboratory values, and genomic information from approximately 6,000 CML patients treated with TKIs from 57 health care organizations. Results: Survival at 20 years was not significantly different between patients with CML treated with TKIs and non-cancer patients (63·4% vs 69·05%, HR 1.095 (0·924 - 1·298) p = 0·295, Figure 1). No significant change in survival probability was seen comparing patients treated 2001-2010 and 2011-2020 (HR 1·113 (0·858 - 1·443), p = 0.42) or comparing imatinib with second-generation TKIs (imatinib vs dasatinib HR 1·017 (0·895 - 1·157), log-rank p = 0·80; imatinib vs nilotinib HR 1·101 (0·925 - 1·310). When comparing the occurrence of second malignancies, an increased incidence was seen in CML patients (64·4% vs 17·2%, OR 8·72 (7·955, 9·549)). In the case of cardiovascular diseases, CML patients again showed a higher incidence than non-CML patients (74·2% vs 60·5%, p<0·001, OR 1·879 (1·729, 2·041)). No specific TKI was associated with the increased risk of any of these conditions. Regarding TKI usage and switching patterns, dasatinib was the most common second-line treatment for both H12O and the US, but not in the EMEA where it was nilotonib. Ponatinib assumed a larger role in third-line and beyond. Image:Summary/Conclusion: From this large real-world analysis, we conclude that in the TKI era, the CML population has the same survival as a non-cancer population. However, the main TKI toxicities and comorbidities remained high in the CML population, and despite them not seeming to impact survival, it could affect the quality of life. Therefore, strategies to stop lifelong TKI use should be prioritized to reduce adverse events.